mRNA-3745

mRNA-3745 is an investigational mRNA-based therapy developed for the treatment of Glycogen Storage Disease Type 1a (GSD1a), also known as von Gierke disease. This rare genetic disorder is characterized by a deficiency in the enzyme glucose-6-phosphatase (G6Pase), leading to impaired glycogen breakdown and severe hypoglycemia, as well as long-term liver and kidney complications. The drug consists of nucleoside-modified, codon-optimized messenger RNA (mRNA) that encodes a modified version of the human G6Pase enzyme (variant S298C). The mRNA is encapsulated within lipid nanoparticles (LNPs) to facilitate delivery into target cells. Once administered intravenously, the LNPs deliver the mRNA into cells where it instructs them to produce functional G6Pase enzyme, potentially correcting the underlying metabolic defect in patients with GSD1a[1][2][3][4][5]. mRNA-3745 has received Orphan Drug Designation from the FDA and is currently being evaluated in phase 1/2 clinical trials for safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy[4][7]. It is being developed by Moderna.

Other names

mRNA encoding human glucose 6-phosphatase variant S298Cmessenger RNA encoding human G6Pase-α S298C